NICE recommends routine NHS access to SMA treatments, offering new hope for patients and families

People living with spinal muscular atrophy (SMA) across England are set to benefit from routine access to potentially life‑changing treatments following final draft guidance from NICE published today (14 May 2026). 

It means everyone with the devastating condition now has a treatment option that is routinely available.  

Evidence gathered from patients receiving nusinersen (also called Spinraza and made by Biogen) and risdiplam (also called Evrysdi and made by Roche Products) has confirmed their benefits. It means everyone with the devastating condition now has a treatment option that is routinely available.    

Today's decision recognises the significant benefits these treatments can bring in improving survival, slowing disease progression and helping people maintain independence and quality of life. 

Both treatments have been available in managed access – nusinersen since 2019 and risdiplam since 2021 – while further evidence on how well they work was collected. 

SMA is a rare, progressive genetic condition that causes severe muscle weakness and can affect breathing, swallowing and movement. Without effective treatment, many people—particularly babies and young children—face profound disability and reduced life expectancy. People with the most severe forms of SMA usually die before the age of 2. It is estimated there are currently around 1,150 people with SMA types 1-3 in England. 

NICE's final draft guidance recommends nusinersen for treating pre-symptomatic and symptomatic SMA that has not been successfully treated with NICE-recommended gene therapy onasemnogene abeparvovec. Risdiplam is recommended for treating SMA types 1, 2 or 3 that has not been successfully treated with onasemnogene abeparvovec and for pre-symptomatic SMA where onasemnogene abeparvovec is not suitable. 

Helen Knight, director of medicines evaluation at NICE, said: “Spinal muscular atrophy is a devastating condition that affects every aspect of daily life for people living with it and for their families. After carefully reviewing the latest evidence and real-world experience from the NHS, our independent committee concluded that nusinersen and risdiplam can offer substantial, life‑changing benefits for many people with SMA. 

“These treatments can help people live longer, maintain their independence, communication and participation in everyday life and reduce the need for hospital care.  

“By recommending routine NHS access to nusinersen and risdiplam, NICE has recognised both the severity of SMA and the transformational impact these treatments can have on people's lives. For patients and families affected by SMA, the guidance represents a major step forward—offering greater certainty, improved quality of life and renewed hope for the future.” 

Public Health Minister, Sharon Hodgson said: “Spinal muscular atrophy can have a profound impact on every aspect of a person's life, particularly for children and young people.

“Today's final draft guidance is welcome news for families across the country and represents an important milestone in improving access to innovative NHS treatments for rare diseases.

“Drawing on both clinical evidence and the real-world experiences of patients already receiving treatment, this decision will help ensure more people with SMA can access the care and support they need to live fuller, more independent lives.”

NICE's evaluation found strong evidence that, compared with best supportive care, nusinersen and risdiplam were shown to reduce the need for permanent ventilation, help children reach key developmental milestones such as sitting, standing and walking, stabilise or improve motor function in children and adults with later onset SMA, slow disease progression and improve survival.  

The final draft guidance also draws on real-world experience from people receiving treatment through NHS managed access schemes. Data from UK SMA registries and patient surveys show that treatment has led to fewer emergency hospital admissions, particularly for respiratory infections, improved physical and mental wellbeing for patients, reduced reliance on unpaid carers, and better participation in education, work and social activities. 

Carers also reported improvements in their own mental health, sleep and ability to balance caring responsibilities with work and family life. 

Importantly, the final draft guidance recognises there is no single “one‑size‑fits‑all” treatment for SMA. It supports access to both nusinersen and risdiplam so that clinicians, patients and families can choose the option that best suits individual needs, preferences and circumstances. 

The treatments differ in how they are given—nusinersen by periodic spinal injection and risdiplam as a daily oral medicine—making choice particularly important for people with complex needs or mobility issues. 

The independent committee heard from patients and clinical experts that even small improvements—such as maintaining hand or arm movement—can have a big impact, allowing people with SMA to use technology, communicate more easily and live more independently.  

The committee also acknowledged that stabilisation of symptoms should be considered a meaningful benefit in a progressive condition like SMA and it supported the use of clinical judgement of a person's individual needs when assessing whether treatment is helping them.  

Nusinersen and risdiplam will be available immediately, with NHS England funding from routine budgets from today. 

ENDS

1. NICE's final draft guidance on nusinersen and risdiplam for treating spinal muscular atrophy is available (from 00:01, 14 May 2026) at: https://www.nice.org.uk/guidance/indevelopment/gid-ta11386/documents